Marinus Pharmaceuticals, Inc. announced the positive preliminary data from the initial CDKL5 patients enrolled in its ongoing Phase 2 open-label study evaluating its CNS-selective GABAA modulator, ganaxolone, as a treatment for orphan, genetic disorders. CDKL5 is a severe, rare genetic disorder that results in early-onset, difficult-to-control seizures, and neuro-developmental impairment. Enrollment is continuing in the study with top-line data expected in mid-2017. Four patients have been enrolled in this cohort of the study and received up to 1,800 mg/kg of ganaxolone per day for an average treatment duration of five months. Three of the four patients experienced a notable reduction in seizure frequency compared to baseline ranging from 52% to 88%. All responders continue to receive treatment, two of whom have completed six months of treatment and have elected to participate in the study extension. One patient discontinued the study after four months of treatment due to lack of efficacy. Safety data to date are consistent with earlier studies where ganaxolone has shown to be generally safe and well-tolerated. The CDKL5 patient showed a >67% seizure reduction and EEG changes consistent with clinical improvement (50% reduction in awake slow-spike wave discharges). The two patients with PCDH19 showed an 80% and 75% reduction in seizure frequency, respectively, and EEG improvement in slow-spike and wave frequency of >90% and 80% on awake and asleep EEG. This Phase 2 open-label trial is currently accepting patients at five sites in the United States and one in Italy. The multi-cohort study is designed to enroll up to 10 patients with each of CDKL5 disorder, Lennox Gastaut Syndrome (LGS) and PCDH19 pediatric epilepsy.